Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease.

The neuropathic glycosphingolipidoses are a subgroup of lysosomal storage disorders for which there are no effective therapies. A potential approach is substrate reduction therapy using inhibitors of glucosylceramide synthase (GCS) to decrease the synthesis of glucosylceramide and related glycosphin...

Täydet tiedot

Bibliografiset tiedot
Päätekijät: Karen M Ashe, Dinesh Bangari, Lingyun Li, Mario A Cabrera-Salazar, Scott D Bercury, Jennifer B Nietupski, Christopher G F Cooper, Johannes M F G Aerts, Edward R Lee, Diane P Copeland, Seng H Cheng, Ronald K Scheule, John Marshall
Aineistotyyppi: Artikkeli
Kieli:English
Julkaistu: Public Library of Science (PLoS) 2011-01-01
Sarja:PLoS ONE
Linkit:https://www.ncbi.nlm.nih.gov/pmc/articles/pmid/21738789/pdf/?tool=EBI